Stanley H. Appel, a world-renowned expert in amyotrophic lateral sclerosis (ALS) and the founder of the first multidisciplinary clinic dedicated to ALS care and research in the U.S., will receive the Tribute Award from the Muscular Dystrophy Association (MDA). The award acknowledges the neurologist’s work in pioneering ALS…
The rarity of amyotrophic lateral sclerosis (ALS) in children makes it impossible to realistically conduct clinical trials of experimental medicines in this population, researchers across Europe argue in a paper. “Given the absence of consensus on pediatric ALS as a disease entity, the extremely low prevalence of ALS in…
After a two-year hiatus, the Muscular Dystrophy Association (MDA) is bringing back its in-person Wings Over Wall Street fundraiser gala in New York City, with proceeds going to support MDA research into promising amyotrophic lateral sclerosis (ALS) treatments. The June 9 event, starting at 6 p.m. EST at…
The Barrow Neurological Institute in Phoenix, Arizona, is calling on people with amyotrophic lateral sclerosis (ALS) and their family members and friends to participate in a biomarker study that aims to create a bank of biological samples and clinical data collected over time. Data will be made available…
One-time delivery of a CRISPR-based gene therapy significantly reduced the buildup of toxic RNA molecules resulting from C9ORF72 mutations in cells and mouse models of amyotrophic lateral sclerosis (ALS), the therapy’s developer, Locanabio, reported. The approach was designed using the company’s CORRECTx platform, which uses CRISPR technology to…
After several years of support, the late-stage biopharmaceutical company Cytokinetics is reaffirming its commitment to the ALS Association and its efforts to battle amyotrophic lateral sclerosis (ALS) and assist the ALS community. In addition to backing nationwide organizational efforts, Cytokinetics has, for more than a decade, provided support…
The estimated 2017 U.S. prevalence of individuals with amyotrophic lateral sclerosis (ALS) was 24,821, or 7.7 per 100,000 people, according to a recent report from the National ALS Registry. While a statistical model was used to account for potentially missing data, the research team emphasized that this number is…
Treatment with AMX0035 significantly prolonged the time that people with amyotrophic lateral sclerosis (ALS) in the CENTAUR clinical trial did not require permanent ventilation or tracheostomy by over seven months, a new study shows. “People living with ALS often require medical interventions like mechanical ventilation as the disease…
Team Gleason and Synchron have entered into a partnership aiming to advance a technology that offers greater accessibility for people with amyotrophic lateral sclerosis (ALS) and other conditions that affect physical movement. Synchron is developing brain computer interfaces, or BCIs — devices that can detect activity in…
A research team has developed a new way to search for genes and gene mutations that may contribute to the development of rare diseases, bypassing some of the challenges typically associated with genetic analyses. For diseases like amyotrophic lateral sclerosis (ALS) in which genetic factors are thought to…
